The proposed research is designed to test the global hypothesis that inhaled corticosteroids (ICS), a therapy developed to treat asthma, will prevent vasoocclusive painful episodes in adults with Sickle Cell Disease (SCD) who wheeze, but do not meet criteria for a diagnosis of asthma. The specific aims of this proposal are 1) Conduct a feasibility study - a randomized controlled trial of ICS for adults with SCD who do not meet criteria for a diagnosis of asthma but report recurrent cough or wheezing, 2) Measure the effects of ICS on biological correlates of pulmonary inflammation (as determined by exhaled nitric oxide) and vascular injury (as determined by sVCAM) in SCD, and 3) Compare properties of traditional and Bayesian adaptive clinical trial design for therapeutic trials in SCD in preparation for designing a definitive trial of ICS. These aims have the potential to 1) change the standard of care for individuals with SCD and recurrent cough or wheeze, 2) provide insight into the pathogenesis of non-asthmatic wheezing in SCD and its response to treatment, 3) explore the suitability of innovative clinical trial designs to overcome the challenges that have hindered therapeutic innovation for SCD.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
54
inhaled cortico-steroid (ICS) with a dosage of 220mcg once daily for 16 weeks
placebo training inhaler with the same instructions as the experimental group.
Icahn School of Medicine at Mount Sinai
New York, New York, United States
Number of Participants Who Completed Follow up
Feasibility is determined by calculating the proportion of randomized participants who complete follow up and a minimum of 30 pain diaries with good adherence to the study medication vs. the number enrolled.
Time frame: at 2 years
Change in Exhaled Nitric Oxide (eNO)
Change in effects of inhaled corticosteroids (ICS) as measured by exhaled nitric oxide levels, which is the primary marker of pulmonary inflammation.
Time frame: Before ICS therapy begins and at 8 weeks post enrollment
Change in Soluble Vascular Cell Adhesion Molecule (sVCAM) Level
Mean Change in effects of inhaled corticosteroids vascular injury, assessed by biomarker sVCAM as a surrogate for vascular injury.
Time frame: Before ICS therapy begins and at 8 weeks post enrollment
Change in Adult Sickle Cell Quality of Life Measurement Information System (ASCQ-Me)
Mean changes in ASCQ-Me (NHLBI developed a patient-reported Sickle Cell Disease (SCD) quality of life measurement tool) pain impact, at week 20 as compared to baseline. A reduction change on a 100-point scale indicated improved quality of life. ASCQ-Me uses a T-score metric (0-100) in which 50 is the mean of the reference population and 10 is the standard deviation (SD) of that population.
Time frame: baseline and week 20
The Medication Adherence Report Scale
The medication adherence report scale for asthma is a 10 question tool scored between 0 and 5, with full scale from 0 to 25, with higher scores indicating greater adherence
Time frame: 20 weeks
Change in the Numerical Rating Scale (NRS) for Pain
Mean change in patient reported pain NRS score, full scale range 0- 10, higher score indicate more pain
Time frame: baseline and 20 weeks
Asthma Control Test
Asthma control test, total score from 0-25, with higher score indicating more symptoms
Time frame: 8 weeks
Admissions or Visits to the Hospital
Number of times participant visited the Emergency Department (ED) or was admitted to the hospital
Time frame: baseline through 8 weeks
Change in Reticulocytes Count
Mean change in reticulocytes count - the number of new red blood cells.
Time frame: baseline and 8 weeks
Change in FEV1/FVC
Mean change in FEV1/FVC at 8 weeks compared to baseline
Time frame: baseline and 8 weeks
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